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METHODS: In this prospective study, severe HA patients were recruited from January 2022 to June 2023. Inhibitor positive and inhibitor negative patients with annual bleeding rate (ABR) 8 or greater and past histories of bleeding like intra-cranial, intra-abdominal, and pseudo-tumors were included. Emicizumab loading dose was 3 mg/kg in the first 4 weeks, and the maintenance dose was started at week 5 at 6 mg/kg/month. Patients' detailed bleeding history and demographics were recorded. The five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) was used to evaluate patients' HRQoL. Furthermore, Hemophilia Joint Health Score (HJHS) and Functional Independence score in Hemophilia (FISH) were applied for the assessment of joints at different time points. Results were analyzed by SPSS version 21. RESULTS: A total of 36 HA male patients with the mean age of 19.7 ± 14.42 years were recruited in the study; among them, 19 patients were inhibitor positive, while 17 were negative. Patients clinically presented with bleeding symptoms which included: hemarthrosis 95%, GI bleeding 13.8%, and bruises and gums bleeding 13.8%. Significant reduction was observed in the bleeding episodes after the therapeutic intervention, and joints assessment and Euro-Quality-of-life Visual Analog Scale showed a significant improvement in health after treatment. Similarly, there was a remarkable reduction in bleeding episodes and improved quality of life among HA patients. The ABR decreased from 53.6% episodes per year prior to treatment to 2.4% during Emicizumab therapy. Prior to initiating Emicizumab therapy, participants exhibited an average FISH score of 16 and HJHS score of 10, indicating moderate limitations due to joint-related issues. After treatment, the mean FISH score improved to 9 and HJHS score to 4 reflecting a substantial enhancement in participants' ability to perform daily activities (P < 0.057). CONCLUSION: Our results showed that HA patients on prophylactic treatment with Emicizumab were less restricted and had improved quality of life due to marked decrease in bleeding episodes which resulted in improved health and social lives. In addition, it was well tolerated, and no participant discontinued treatment because of adverse events.

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OBJECTIVES: We aimed to discuss the correlation between the Hemophilia Early Detection Ultrasound in China (HEAD-US-C) score and the Hemophilia Joint Health Score version 2.1 (HJHS 2.1) of the knee joint in patients with hemophilia. METHODS: We included 70 male patients with hemophilia admitted to The Second Hospital of Shanxi Medical University; the patients' bilateral knee joints were evaluated using the HEAD-US-C score and HJHS. We analyzed factors influencing hemophilia arthropathy of the knee and examined the correlation between the HEAD-US-C score and HJHS. RESULTS: The joint injury severity was positively correlated with age and the number of bleeds (P < .001). Further, the HEAD-US-C score and HJHS differed according to the severity (both P < .001), but not type (P = .163 and P = .283, respectively), of hemophilia. There was a significant correlation between the HEAD-US-C score and HJHS (P < .001). CONCLUSIONS: Overall, all joint lesions observed on ultrasound corresponded to clinical joint functional abnormalities. Therefore, the HEAD-US-C is important for hemophilic arthropathy evaluation and is useful in explaining abnormal joint function.

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AIM: To screen types of behavioral problems among children with hemophilia and their relation to the disease parameters. METHODS: Fifty boys, 6-18 years old, with moderate and sever hemophilia were subjected to: history taking, joint evaluation using the Hemophilia Joint Health Score, and behavioral assessment using the Child Behavioral Check List. RESULTS: Patients experienced different patterns of behavior disorder. Patients' age significantly correlated with anxious/depressed behavior, somatic complaints, social problems, aggressive behavior, internalizing behavior, and total behavior problems. Hemophilia severity significantly correlated with social problems, thought problems, aggressive behavior, internalizing behavior, externalizing behavior, and total behavior disorders. Affected joint number significantly correlated with withdrawn/depressed behavior, social problems, thought problems, aggressive behavior, internalizing behavior, and total behavior disorders. A high Hemophilia Joint Health Score of the target joints was significantly correlated with social problems, rule-breaking behavior, aggressive behavior, externalizing behavior, and total behavior disorders. Hemophilic arthropathy duration significantly correlated with somatic complaints, social problems, thought problems, attention problems, aggressive behavior, internalizing behavior, externalizing behavior, and total behavior problems. CONCLUSION: Children with hemophilia had behavioral disorders. The most affected scale was aggressive behavior. The least affected scale was attention problems. Behavioral disorders in children with hemophilia are influenced by the age of the patient, the severity of the disease, the number of joints affected, the duration of hemophilic arthropathy, and the score of joint affected by Hemophilia Joint Health Score.

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OBJECTIVES: To explore the long-term joint outcomes of low-dose prophylaxis in Chinese children with severe haemophilia A and to analyse their related factors. METHODS: We retrospectively analysed follow-up data from 21 severe haemophilia A children on regular low-dose prophylaxis for 6-10 years. We used International Prophylaxis Study Group magnetic resonance imaging score (IPSG MRI score), Hemophilia Joint Health Score (HJHS), number of target joints, and Hemophilia-Specific Quality of Life Index (Haemo-QoL) to evaluate joint outcomes. Factors associated with these outcomes were evaluated by statistical analysis. RESULTS: (1) The children were 1.75 to 17 years age at prophylaxis initiation. Median prophylactic factor VIII dose was 22.9 IU/kg per week. (2) At the end of follow-up: (a) The total IPSG MRI scores were 2-24 with 90.5% children exhibiting moderate to severe joint involvement (score 7-24); (b) The HJHS ranged 2-27, with 0-10 for 46.7% children and >10 for 53.3% children. There was a positive correlation between the MRI score and HJHS (p < .05); (c) Compared to their on-demand treatment period before prophylaxis, target joints numbers decreased, and no child needed auxiliary devices to walk; (d) Joint outcomes were positively correlated with the age at initiation of low-dose prophylaxis (p < .05) and negatively correlated with the treatment dose. CONCLUSION: Long-term low-dose prophylaxis had positive effect on joint outcomes compared with on-demand treatment. However, a certain degree of joint damage remained in all children indicating the need for improving the current strategy of low-dose prophylaxis.

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BACKGROUND: In countries with limited resource, haemophilia patients have to choose low-dose prophylaxis or on-demand treatment (ODT) because of economic constraints. Whether low-dose prophylaxis can achieve better joint function outcome than ODT over long-term remains unclear. AIM: To investigate the long-term effect of low-dose tertiary prophylaxis versus ODT on joint health in severe haemophilia A children. METHODS: This was a retrospective study. We enrolled and followed 34 severe haemophilia boys in China receiving on-demand treatment (n = 18) or low-dose prophylaxis (10-15 IU/kg, 2-3 times per week) for a medium-term (6-18 months, n = 9) or longer-term (19-30 months, n = 7). We evaluated their haemophilia joint health score (HJHS) 2.1 and functional independence score in haemophilia (FISH) at baseline and at their 6-year follow-up visits. Their annual bleeding rate (ABR) and annual joint bleeding rate (AJBR) were also recorded. RESULTS: During the 6-year follow-up period, ABR and AJBR were similar between the 2 prophylaxis groups, with each of the 2 prophylaxis group rates being significantly better (lower) than the ODT group (P < .05). Compared to baseline values, evaluation at 6-year follow-up showed HJHS improvement in both prophylaxis groups, although significantly (P < .05) only in the longer-term prophylaxis group. The FISH score showed insignificant change in patients in each prophylaxis cohort, compared to significant worsening (P < .05) in the ODT group. CONCLUSION: Low-dose tertiary prophylaxis reduced ABR and AJBR of children with severe haemophilia and better maintained their functional independence by the FISH over the long term. Longer-term prophylaxis also improved their joint health status by the HJHS.

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Prophylaxis is the gold standard treatment for haemophilia but requires more amount of clotting factor concentrates, than on demand therapy. Low dose prophylaxis is an alternative for countries with limited resources. There are data of evidence showing the superiority of low-dose prophylaxis than episodic treatment. Studies from China, India, Tunisia, Thailand and Indonesia reported experiences with low dose prophylaxis using outcome assessment. These studies have shown the effectiveness of various protocols regimen with once, twice or thrice injection of 10-15 UI Kg-1 per injection. These protocols allow reduction of joint bleeds and at least delay of joint damages. There is not enough long-term data nowadays, but low dose prophylaxis is certainly better than on demand therapy and should be considered as a first step of prophylaxis in some countries but not the final goal.

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Among different groups of hemophiliacs, those suffering from Severe Hemophilia A (SHA) are most vulnerable to the complications of the disease. This study investigated the Health-Related Quality of Life (HR-QoL) among adult patients with SHA. A cross-sectional study was designed to gather demographic and clinical information from adult patients with SHA. Patients with inhibitors were excluded. The remaining were asked to complete the HR-QoL questionnaire after being examined for joint health using the Hemophilia Joint Health Score (HJHS). The HR-QoL and joint conditions were measured in 38 patients. The mean EQ-5D value scores were 0.46 (SD=0.23) while the mean Visual Analogous Scale score was 50 (SD=18.7). The clinical examination of patients indicated that the HJHS were as follows: eight patients had a score of 55-75, 12 patients had a score of 40-55, 7 of them (25-40) and 11 patients had a score of 10-25. The results obtained from this study showed that HRQoL in hemophilia patients was considerably low. Pain, anxiety/depression, and motion limitations were the main causes of the disutility for these patients respectively.

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INTRODUCTION: Evidence suggests that toxic iron is involved in haemophilic joint destruction. AIM: To determine whether joint iron deposition is linked to clinical and imaging outcomes in order to optimize management of haemophilic joint disease. METHODS: Adults with haemophilia A or haemophilia B (n = 23, ≥ age 21) of all severities were recruited prospectively to undergo assessment with Hemophilia Joint Health Scores (HJHS), pain scores (visual analogue scale [VAS]) and magnetic resonance imaging (MRI) at 3T using conventional MRI protocols and 4-echo 3D-UTE-Cones sequences for one affected arthropathic joint. MRI was scored blinded by two musculoskeletal radiologists using the International Prophylaxis Study Group (IPSG) MRI scale. Additionally, UTE-T2* values of cartilage were quantified. Correlations between parameters were performed using Spearman rank correlation. Two patients subsequently underwent knee arthroplasty, which permitted linking of histological findings (including Perl's reaction) with MRI results. RESULTS: MRI scores did not correlate with pain scores or HJHS. Sixteen joints had sufficient cartilage for UTE-T2* analysis. T2* values for cartilage correlated inversely with HJHS (rs  = -0.81, P < 0.001) and MRI scores (rs  = -0.52, P = 0.037). This was unexpected since UTE-T2* values decrease with better joint status in patients with osteoarthritis, suggesting that iron was present and responsible for the effects. Histological analysis of cartilage confirmed iron deposition within chondrocytes, associated with low UTE-T2* values. CONCLUSIONS: Iron accumulation can occur in cartilage (not only in synovium) and shows a clear association with joint health. Cartilage iron is a novel biomarker which, if quantifiable with innovative joint-specific MRI T2* sequences, may guide treatment optimization.

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Hemophilia refers to a group of bleeding disorders in which there is a deficiency of one of the factors necessary for coagulation of the blood. Susceptibility to joint hemorrhage in persons with Hemophilia suggests that the routine assessment of joint health is an important aspect of clinical management and outcome studies assessing the efficacy of treatment. This prospective study was conducted to study joint health status in Hemophilia patients and draw their joint disability score by using Hemophilia Joint Health Score (HJHS). Out of total 56 cases 51 (91.07 %) cases were diagnosed as hemophilia A while 5 cases (8.92 %) were diagnosed as hemophilia B. According to their factor level 44 % cases had severe 36 % had moderate and 20 % had mild disease. Knee joint was the predominant joint affected by hemarthrosis in 67.85 % cases followed by ankle joint (51.7 %) elbow joint (35.7 %), hip joint (12.5 %), shoulder joint (5.3 %) and proximal metacarpophalangeal joint (1.78 %).Out of total 37.5 % patients of hemophilia had developed target joint. Knee joint was the predominant target joint in 28.57 % cases and ankle joint was the target joint in 8.92 % cases. Maximum number of patients (40.47 %) had HJHS score of zero. The mean HJHS score was 6.78 ± 9.04. HJHS score showing significant positive correlation with age of patient (p < 0.0001). Most risky period and most aggravating development of hemophilic joint damage starts from 7 years of age. Therefore, treatment decisions, such as starting prophylaxis, should be tailored according to bleeding pattern and age of patients rather than based on the clotting factor activity levels.